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Gene therapy for rare immune disease shows promise in Long-Term study

NCT ID NCT06282432

Disease control Sponsor: Rocket Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 9 people with a rare immune disorder called LAD-I who received a one-time gene therapy that adds a working copy of the ITGB2 gene to their own blood stem cells. Researchers want to see if the treatment safely controls the disease over many years and helps patients avoid a bone marrow transplant. The study tracks survival without transplant, infections, hospital stays, and skin rashes.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
gene therapy (modified stem cells with a working ITGB2 gene)
What this could lead to
If successful, this could show that a one-time gene therapy safely controls LAD-I for years, avoiding the need for a bone marrow transplant.
What could go wrong
This is a very small follow-up study (9 people) with no comparison group, so results may not apply to everyone. Long-term risks of gene therapy are still unknown.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

9 people

The number who actually took part.

Started

Mar 2022

Expected to finish

Oct 2036

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Subjects that have been treated with RP-L201 on the RP-L201-0318 study.

Ages

3 months and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Enrolled in the Phase I/II Study RP-L201-0318. 2. Received an autologous infusion of CD34+ hematopoietic stem cells modified with a lentiviral vector containing the ITGB2 gene, encoding for the human CD18 receptor in the parent Study RP-L201-0318. 3. Able to adhere to the study visit schedule and other protocol requirements. 4. Provided written informed consent and, as applicable, assent to participate in the current study. Exclusion Criteria: There are no criteria for exclusion in this study.

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Conditions

The condition(s) this trial relates to.

leukocyte adhesion deficiency leukocyte adhesion deficiency 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Leukocyte adhesion deficiency

Contacts and locations

Locations

  • Hospital Infantil Universitario Niño Jesús

    Madrid, 28009, Spain

  • University College London Great Ormond Street Institute of Child Health (GOSH)

    London, WC1N 1EH, United Kingdom

  • University of California, Los Angeles (UCLA)

    Los Angeles, California, 90095-1489, United States

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